Retinal HemangioblastomaNCT04458935
Retrospective Case Series of Trans-scleral Cryotherapy for Retinal Hemangioblastoma
An observational study of Retinal Hemangioblastoma and Von Hippel-Lindau Disease, run by National Eye Institute (NEI) at 1 site in United States.
Trial summary
Background: Retinal hemangioblastoma (RH) is a tumor. It grows from the retina in the eye. It can threaten a person s vision. Trans-scleral cryotherapy is used to destroy the tumors and minimize the long-term risks of vision loss. RH is a rare condition, often occurring in people with von Hippel-Lindau disease. Read more about this study
- Who is it for?
- Ages 7 to 120 years
- Conditions the study is for
- Retinal Hemangioblastoma, Von Hippel-Lindau Disease
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Jul 2020to 2 Jun 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 120 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Data (medical history and images) was collected under NEI and non-NEI natural history protocols allowing administration of standard care measures to participants.
Who may be able to join
- Participants were enrolled in NEI and non-NEI natural history protocols, but in all cases, cryotherapy was administered as a standard care measure (not as an investigative treatment).
- Patients were identified by searching the NIH eye clinic EMR for all of those manifesting one or more retinal hemangioblastomas (with or without associated von Hippel-Lindau disease) treated with cryotherapy.
Who cannot join
- None
Trial locations and contacts
1 site in 1 country.
United States1 site
National Eye Institute (NEI)
Bethesda, Maryland, 20892
About this study
Background:
Retinal hemangioblastoma (RH) is a tumor. It grows from the retina in the eye. It can threaten a person s vision. Trans-scleral cryotherapy is used to destroy the tumors and minimize the long-term risks of vision loss. RH is a rare condition, often occurring in people with von Hippel-Lindau disease. There are no clinical trials to study how well the treatment works. Researchers want to study the medical records of people with RH who were treated at the NIH eye clinic to learn more.
Read more
Objective:
To analyze clinical data collected over a 20-year span to study consecutive cases of RH managed with trans-scleral cryotherapy at the NIH.
Eligibility:
People who took part in NIH natural history protocols for which cryotherapy of RH was performed as a standard care measure.
Design:
Researchers will collect and study data from participants medical charts. Participants will not be contacted because no new data is needed. Researchers were granted a waiver of informed consent for use of these medical records. To protect patient privacy, participants will be assigned an ID number. Their data will be entered into a spreadsheet in a coded fashion. The key to this code will be kept in a secure file. No patient identifying information will be used in the analysis or the publication....
Read the detailed description
Study Description: This study is a retrospective review of medical records in the NIH eye clinic. To our knowledge, there has been no report in the current era dedicated to a description of trans-scleral cryotherapy for retinal hemangioblastomas despite its routine use in this setting. This study aims to explore features and outcomes of these cases to better inform best practices.
Objectives: The objective of this study is to perform a retrospective analysis of consecutive cases of retinal hemangioblastoma managed with trans-scleral cryotherapy at the NIH (single center retrospective case series).
- Keywords
- Vascular Tumor, Vision Loss, Natural History
- Medical subject headings
- von Hippel-Lindau Disease, Vascular Neoplasms, Vision Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Participants | Participants with retinal hemangioblastoma (RH) managed with trans-scleral cryotherapy at the NIH. |
What is being measured
The primary outcome is the main question the study is designed to answer.
To perform a retrospective analysis of consecutive cases of retinal hemangioblastoma (RH) managed with trans-scleral cryotherapy at the NIH
Some key measures of interest will include treatment outcomes (rate of control of tumors; visual function measures; post-treatment lesion characteristics; sequelae and complications) and association of outcomes with eye and lesion characteristics.
Measured: ongoing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2020
- Study start2 Jul 2020
- Primary completion2 Jun 2026
- Study completion2 Jun 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinal HemangioblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Eye Institute (NEI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Emily Y Chew, M.D., Principal investigator, National Eye Institute (NEI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04458935
- Sponsor's protocol ID
- 999920129
- Other IDs
- 20-EI-N129
- Status last verified
- Jun 2026
- First submitted
- 3 Jul 2020
- Last updated
- 10 Jun 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Case Series of Trans-scleral Cryotherapy for Retinal Hemangioblastoma
- ClinicalTrials.gov ID
- NCT04458935
- Acronym / sponsor ID
- 999920129
- Keywords
- Vascular Tumor, Vision Loss, Natural History
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- National Eye Institute (NEI)US National Institutes of Health
- Study start
- 2 Jul 2020 (actual)
- Primary completion
- 2 Jun 2026 (actual)
- Study completion
- 2 Jun 2026 (actual)
- First posted
- 7 Jul 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
Retinal hemangioblastoma (RH) is a tumor. It grows from the retina in the eye. It can threaten a person s vision. Trans-scleral cryotherapy is used to destroy the tumors and minimize the long-term risks of vision loss. RH is a rare condition, often occurring in people with von Hippel-Lindau disease. There are no clinical trials to study how well the treatment works. Researchers want to study the medical records of people with RH who were treated at the NIH eye clinic to learn more.
Objective:
To analyze clinical data collected over a 20-year span to study consecutive cases of RH managed with trans-scleral cryotherapy at the NIH.
Eligibility:
People who took part in NIH natural history protocols for which cryotherapy of RH was performed as a standard care measure.
Design:
Researchers will collect and study data from participants medical charts. Participants will not be contacted because no new data is needed. Researchers were granted a waiver of informed consent for use of these medical records. To protect patient privacy, participants will be assigned an ID number. Their data will be entered into a spreadsheet in a coded fashion. The key to this code will be kept in a secure file. No patient identifying information will be used in the analysis or the publication....
Detailed description
Study Description: This study is a retrospective review of medical records in the NIH eye clinic. To our knowledge, there has been no report in the current era dedicated to a description of trans-scleral cryotherapy for retinal hemangioblastomas despite its routine use in this setting. This study aims to explore features and outcomes of these cases to better inform best practices.
Objectives: The objective of this study is to perform a retrospective analysis of consecutive cases of retinal hemangioblastoma managed with trans-scleral cryotherapy at the NIH (single center retrospective case series).
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants | – | –Participants with retinal hemangioblastoma (RH) managed with trans-scleral cryotherapy at the NIH. |
Who may qualify
- Ages
- 7 to 120 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Data (medical history and images) was collected under NEI and non-NEI natural history protocols allowing administration of standard care measures to participants.
Key inclusion criteria (2 listed)
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To perform a retrospective analysis of consecutive cases of retinal hemangioblastoma (RH) managed with trans-scleral cryotherapy at the NIH
Time frame: ongoing
Some key measures of interest will include treatment outcomes (rate of control of tumors; visual function measures; post-treatment lesion characteristics; sequelae and complications) and association of outcomes with eye and lesion characteristics.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Emily Y Chew, M.D. principal investigator
National Eye Institute (NEI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Retinal Hemangioblastoma and Von Hippel-Lindau Disease, run by National Eye Institute (NEI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To perform a retrospective analysis of consecutive cases of retinal hemangioblastoma (RH) managed with trans-scleral cryotherapy at the NIH (time frame: ongoing). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jun 2026.